Branched Poly(-amino esters) for the Delivery of Nucleic Acids

This invention is a formula for the creation of branched poly(β-amino esters) (PBAEs). Branched PBAEs are polymers designed for the efficient delivery of biomolecules into cells. The branched structure of these polymers, created by reacting primary amines with diacrylates especially enhances their ability to deliver nucleic acids by providing better encapsulation and protection during delivery compared to traditional linear polymers. This advancement in nucleic acid delivery technology opens new possibilities for gene therapy and other medical applications where precise delivery of genetic material is necessary. 

Researchers

Giovanni Traverso / Yunhua Shi / Ameya Kirtane / Grace Junyue Zhong / Daniel Reker / Robert Langer / Hyunjoon Kim / Netra Kamat / James Byrne / Gary Liu

Departments: Department of Mechanical Engineering, David H Koch Institute for Integrative Cancer Res, Office of the Institute Professors
Technology Areas: Biotechnology: DNA & RNA Editing / Drug Delivery: Microparticles & Nanoparticles / Therapeutics: Nucleic Acids, Small Molecules
Impact Areas: Healthy Living

  • branched poly(-amino esters) for the delivery of nucleic acids
    United States of America | Published application
  • branched poly(-amino esters) for the delivery of nucleic acids
    European Patent Convention | Published application

Technology 

The technology involves synthesizing branched PBAEs by linking different linear PBAE polymers with branched linkers. This process begins with the reaction of primary amines and diacrylate monomers to form linear PBAEs. These linear polymers are then connected via branched linkers, resulting in branched hybrid polymers. The branched structure enhances the ability of these polymers to complex with and deliver nucleic acids into cells more effectively, improving transfection efficiency and biocompatibility. Furthermore, branched PBAEs can be optimized to deliver proteins, peptide, polynucleotides, vaccine, and immunological agents into cells. 

Problem Addressed 

This technology addresses the challenge of efficiently delivering nucleic acids into the cells, which is hindered by the large size and charge of nucleic acids. Traditional delivery methods, such as naturally occurring polymers like poly(lysine) and synthetic polymers like poly(ethyleneimine), suffer from poor transfection efficiency and associated toxicities. Branched PBAEs overcome these limitations by offering higher transfection efficiency and better biocompatibility compared to these traditional polymers. 

Advantages 

  • These polymers are biodegradable, and less toxic compared to traditional synthetic polymers
  • Branched PBAEs deliver nucleic acids more effectively than linear polymers
  • The ability to modify building blocks allows for optimization of polymer properties to deliver different types of biomolecules

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